Drug Type Small molecule drug |
Synonyms Branaplam Hydrochloride, LMI 070, LMI-070 + [2] |
Target |
Mechanism SMN1 modulators, SMN2 modulators(Survival motor neuron protein modulators) |
Therapeutic Areas |
Active Indication |
Inactive Indication |
Originator Organization |
Active Organization |
Inactive Organization |
Drug Highest PhasePhase 1/2 |
First Approval Date- |
RegulationOrphan Drug (US), Fast Track (US), Orphan Drug (EU) |
Molecular FormulaC22H27N5O2 |
InChIKeySTWTUEAWRAIWJG-UHFFFAOYSA-N |
CAS Registry1562338-42-4 |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Huntington Disease | Phase 2 | CA | 08 Dec 2021 | |
Huntington Disease | Phase 2 | FR | 08 Dec 2021 | |
Huntington Disease | Phase 2 | DE | 08 Dec 2021 | |
Huntington Disease | Phase 2 | HU | 08 Dec 2021 | |
Huntington Disease | Phase 2 | ES | 08 Dec 2021 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 2 | BE | 02 Apr 2015 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 2 | BG | 02 Apr 2015 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 2 | DK | 02 Apr 2015 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 2 | DE | 02 Apr 2015 | |
HMN (Hereditary Motor Neuropathy) Proximal Type I | Phase 2 | IT | 02 Apr 2015 |